Haemophilia ASynoPrevNCT06352216
Prevalence of Synovitis in Patients With Haemophilia A
An observational study of Haemophilia A and Synovitis, run by University Hospital, Bonn at 1 site in Germany.
Trial summary
The aim of this study is to evaluate the prevalence of synovitis in adult patients with haemophilia A. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Haemophilia A, Synovitis, Hemophilia Arthropathy
- What is being tested?
- Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Haemophilia joint health score (HJHS)
- How many people
- 300 participants
- Where
- 1 siteGermany
- Study dates
- 15 Apr 2024to Mar 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients (≥ 18 years of age) suffering from moderate (FVIII 1-5 %) or severe (FVIII \<1 %) haemophilia A.
Who may be able to join
- Participants (≥ 18 years old) suffering from moderate or severe haemophilia A, also with inhibitor or joint replacement
- Complete documentation of hemophilia severity, treatment history (past 12 months), and bleeding events (past 12 months), inhibitor status (at time of enrollment and in past medical history)
- Submitted written consent to participate in the study
Who cannot join
- Patients suffering from other bleeding disorders conflicting with the research question
- Patients in clinical studies with investigational drugs
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Germany1 site, 1 recruiting
- Recruiting
University Hospital Bonn
Bonn, North Rhine-Westphalia, 53113
Andreas C. Strauss, PD Dr. med., 004922828714176, andreas.strauss@ukbonn.de
Andreas C. Strauss, PD Dr. med.
About this study
Official title: Prevalence of Synovitis in Adult Patients With Haemophilia A in Germany and Austria
The aim of this study is to evaluate the prevalence of synovitis in adult patients with haemophilia A.
Read the detailed description
The bleeding disorder haemophilia causes recurrent spontaneous bleedings primarily into the joints and soft tissues. The reason is a congenital lack of factor VIII (haemophilia A) or factor IX (haemophilia B). In Germany, the most affected joints are the ankle, knee, and elbow. The joint bleedings are accompanied by pain and an inflammatory process that leads to synovitis and ultimately to degenerative joint changes resulting in haemophilic arthropathy: The synovial tissue is responsible for removing blood residuals from the joint cavity. However, blood components, especially iron, induce several changes in the synovial tissue. The resulting synovitis leads to damage of cartilage, bone cysts, osteophytes, and joint effusions. Synovitis can even become a bleeding-independent process, especially after repeated bleeding. According to current knowledge, there is no option to prevent synovitis except for preventing bleedings.
In the aftermath of recurrent joint bleedings, various changes in the synovial tissue, the articular cartilage, and the subchondral bone and blood vessels are observed. These changes are accompanied by restricted joint mobility and musculoskeletal dysfunction. In addition to these peripheral structural changes, various studies show that the entire musculoskeletal system is also affected by recurrent joint bleedings, as it adapts to pain.
Improved therapy over the past decades has led to a reduction in major joint bleeding rates. For this reason, it is important to examine whether this has improved the average joint status and how often patients with haemophilia (PwH) are affected by synovitis at all. Sonography is the radiation-free method of choice for the initial determination of synovitis.
In addition to the structural consequences, joint bleedings also lead to pain and reduction in physical performance. When considering the overall process of degenerative joint changes of PwH, it becomes apparent that this also reduces daily mobility and muscle strength. To the best of our knowledge, the effects of the degree of synovitis on pain perception and physical performance have not been evaluated in previous studies.
For this reason, the prevalence of synovitis in PwH and the consequences in relation to the pain situation and physical performance will be investigated.
- Conditions
- Haemophilia A, Synovitis, Hemophilia Arthropathy, Sonography
- Being tested
- Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Haemophilia joint health score (HJHS)
- Keywords
- Synovitis, Haemophilia A, Prevalence
- Medical subject headings
- Hemophilia A, Synovitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Haemophilia joint health score (HJHS)Diagnostic test
Each subject is examined for the presence of synovitis using the aforementioned diagnostics.
| Study group | Role | What they receive |
|---|---|---|
| Patients with Haemophilia A | Patients with severe and moderate Haemophilia A (FVIII \< 5 %, ≥ 18 years' old) Receives: Diagnostic Test: Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Haemophilia joint health score (HJHS) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Haemophilia early arthropathy detection with ultrasound (HEAD-US)
HEAD-US protocol examines both elbow, knee and ankle joints sonographically and assesses the presence of synovitis or joint damage from 0-8 points per joint according to Martinoli et al. 2013. A "total score" of 0 to 48 points is achievable. A higher score means a worse joint status.
Measured: 20 minutes
Secondary outcomes (3)
The Hemophilia Joint Health Score version 2.1 (HJHS)
Clinical joint examination according to De la Corte-Rodriguez et al. 2020. HJHS protocol examines both elbow, knee and ankle joints using a goniometer for range of motion and axial alignment (0-3 points), pain numerical rating scale (0-10 points), swelling (0-3 points), crepitation (0-2 points) and gait abnormalities (0-4 points). A "total score" of 0 to 124 points is achievable. A higher score means a worse joint status.
Measured: 45 minutes
PROMIS-29 Questionaire
Patient-reported outcomes are surveyed using short questionnaires with a stable number of items from seven PROMIS sub scales ("depression", "anxiety", "physical functioning", "impairment due to pain", "fatigue", "sleep impairment", "participation in social roles" and "activities"). The measures use a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population.
For the sub scales "participation in social roles and activities" and "physical functioning", a high score is interpreted as a good result and for the sub scales "anxiety", "depression", "fatigue", "impairment due to pain", and "sleep impairment" a low score is a good result.
Measured: 10 minutes
Haemophilia Activities List (HAL)
to evaluate to subjective physical performance. A higher scores represent a better functional status.
Scores can be calculated for each of ten domains of the HAL and a sum score ("Lying / sitting / kneeling / standing" (8-48 Points), "Functions of the legs" (9-54 Points), "Functions of the arms" (4-24 Points), "Use of transportation" (3-18 Points), "Self care" (5-30 Points), "Household tasks" (6-36 Points), "Leisure activities and sports" (7-42 Points), "Upper Extremity Activities" (9-54 Points), "Basic Lower Extremity Activities" (6-36 Points), "Complex Lower Extremity Activities" (9-54 Points), and "Sum score" (42-252 Points).
Measured: 10 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2024
- Study start15 Apr 2024
- Primary completionFeb 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Bonn
- Sponsor type
- University, hospital or other
- Collaborators
- Swedish Orphan Biovitrum
- Responsible party
- Principal investigator, PD Dr. Andreas Strauß, University Hospital, Bonn
- Investigators
- Andreas C Strauss, PD Dr. med., Principal investigator, University Hospital, Bonn
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06352216
- Sponsor's protocol ID
- Elo-Ger-2023-1642
- Acronym
- SynoPrev
- Status last verified
- Apr 2024
- First submitted
- 7 Mar 2024
- Last updated
- 16 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The publication of anonymous patient data is not allowed by informed consent.
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prevalence of Synovitis in Adult Patients With Haemophilia A in Germany and Austria
- ClinicalTrials.gov ID
- NCT06352216
- Acronym / sponsor ID
- SynoPrev / Elo-Ger-2023-1642
- Conditions
- Haemophilia A, Synovitis, Hemophilia Arthropathy, Sonography
- Keywords
- Synovitis, Haemophilia A, Prevalence
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- University Hospital, BonnUniversity, hospital or other
- Collaborators
- Swedish Orphan Biovitrum
- Study start
- 15 Apr 2024 (actual)
- Primary completion
- Feb 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 8 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to evaluate the prevalence of synovitis in adult patients with haemophilia A.
Detailed description
The bleeding disorder haemophilia causes recurrent spontaneous bleedings primarily into the joints and soft tissues. The reason is a congenital lack of factor VIII (haemophilia A) or factor IX (haemophilia B). In Germany, the most affected joints are the ankle, knee, and elbow. The joint bleedings are accompanied by pain and an inflammatory process that leads to synovitis and ultimately to degenerative joint changes resulting in haemophilic arthropathy: The synovial tissue is responsible for removing blood residuals from the joint cavity. However, blood components, especially iron, induce several changes in the synovial tissue. The resulting synovitis leads to damage of cartilage, bone cysts, osteophytes, and joint effusions. Synovitis can even become a bleeding-independent process, especially after repeated bleeding. According to current knowledge, there is no option to prevent synovitis except for preventing bleedings.
In the aftermath of recurrent joint bleedings, various changes in the synovial tissue, the articular cartilage, and the subchondral bone and blood vessels are observed. These changes are accompanied by restricted joint mobility and musculoskeletal dysfunction. In addition to these peripheral structural changes, various studies show that the entire musculoskeletal system is also affected by recurrent joint bleedings, as it adapts to pain.
Improved therapy over the past decades has led to a reduction in major joint bleeding rates. For this reason, it is important to examine whether this has improved the average joint status and how often patients with haemophilia (PwH) are affected by synovitis at all. Sonography is the radiation-free method of choice for the initial determination of synovitis.
In addition to the structural consequences, joint bleedings also lead to pain and reduction in physical performance. When considering the overall process of degenerative joint changes of PwH, it becomes apparent that this also reduces daily mobility and muscle strength. To the best of our knowledge, the effects of the degree of synovitis on pain perception and physical performance have not been evaluated in previous studies.
For this reason, the prevalence of synovitis in PwH and the consequences in relation to the pain situation and physical performance will be investigated.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with Haemophilia A | – | Diagnostic Test: Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Haemophilia joint health score (HJHS)Patients with severe and moderate Haemophilia A (FVIII \< 5 %, ≥ 18 years' old) |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult patients (≥ 18 years of age) suffering from moderate (FVIII 1-5 %) or severe (FVIII \<1 %) haemophilia A.
Key inclusion criteria (3 listed)
- Participants (≥ 18 years old) suffering from moderate or severe haemophilia A, also with inhibitor or joint replacement
- Complete documentation of hemophilia severity, treatment history (past 12 months), and bleeding events (past 12 months), inhibitor status (at time of enrollment and in past medical history)
- Submitted written consent to participate in the study
Key exclusion criteria (2 listed)
- Patients suffering from other bleeding disorders conflicting with the research question
- Patients in clinical studies with investigational drugs
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Haemophilia joint health score (HJHS)Each subject is examined for the presence of synovitis using the aforementioned diagnostics. | Diagnostic test | Patients with Haemophilia A |
Primary outcome
Haemophilia early arthropathy detection with ultrasound (HEAD-US)
Time frame: 20 minutes
HEAD-US protocol examines both elbow, knee and ankle joints sonographically and assesses the presence of synovitis or joint damage from 0-8 points per joint according to Martinoli et al. 2013. A "total score" of 0 to 48 points is achievable. A higher score means a worse joint status.
3 secondary outcomes
The Hemophilia Joint Health Score version 2.1 (HJHS)
Time frame: 45 minutes
PROMIS-29 Questionaire
Time frame: 10 minutes
Haemophilia Activities List (HAL)
Time frame: 10 minutes
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2024.
- Individual participant dataSharing planned: No. The publication of anonymous patient data is not allowed by informed consent.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Andreas C Strauss, PD Dr. med. principal investigator
University Hospital, Bonn
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Haemophilia A and Synovitis, run by University Hospital, Bonn at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Andreas C. Strauss, PD Dr. med. (+4922828714176, andreas.strauss@ukbonn.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Haemophilia early arthropathy detection with ultrasound (HEAD-US) (time frame: 20 minutes). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2024.